Industry Updates
Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
This week, Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, […]
Read More ›Genentech Releases SMA Community Letter
Our industry partners at Genentech recently released an SMA community letter summarizing 2022 developments and initiates. Check it out here!
Read More ›Biogen Releases Q3 SMA Community Statement
Learn about Biogen’s latest news in SMA by viewing their Community Statement and a related video.
Read More ›Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 […]
Read More ›New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which […]
Read More ›SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
Read More ›SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
Read More ›Community Statement from Novartis Gene Therapies: Zolgensma Safety Monitoring Update on Acute Liver Failure
Dear SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of […]
Read More ›Biogen Shares Community Statement for SMA Awareness Month
Learn about Biogen’s latest news in SMA by viewing their Community Statement (available as a PDF) and a related video.
Read More ›SMA Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
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